A Study of FCX-013 Plus Veledimex for the Treatment of Moderate to Severe Localized Scleroderma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government.
Know the risks and potential benefits of clinical studies and talk to your health care provider before participating.
Read our disclaimer for details.

A two-component therapeutic consisting of FCX-013 and veledimex for the treatment of localized scleroderma (or morphea). The first component, FCX-013, is autologous human fibroblasts genetically-modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), a protein responsible for breaking down collagen. FCX-013 is designed to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. With the FCX-013 therapy, the patient will take an oral compound (Veledimex) to induce MMP-1 protein expression from the injected cells. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further MMP-1 production from the injected cells. FCX-013 plus veledimex is being developed in anticipation of improving skin function in patients by resolving fibrotic lesions and normalizing dermal collagen production

Condition or disease

Intervention/treatment

Phase

MorpheaScleroderma, LocalizedScleroderma

Genetic: FCX-013Drug: veledimex

Phase 1Phase 2

Detailed Description:

Fibrocell is developing a two-component therapeutic product for the treatment of moderate to severe localized scleroderma (morphea). The first component is FCX-013, an autologous genetically modified human dermal fibroblast (GM-HDFs) cell product that is genetically modified with a lentiviral vector (LV) to express human matrix metalloproteinase 1 (MMP1). MMP1 is also known as interstitial collagenase or fibroblast collagenase and is an enzyme that under normal physiological conditions breaks down the extracellular matrix. Specifically, MMP1 breaks down interstitial collagens, types I, II and III. In addition, the FCX-013 cells are also transduced with genetic constructs that encode the RheoSwitch Therapeutic System® (RTS®) an inducible promotor system that in the presence of a small molecule activator ligand controls expression of the MMP1 gene. The RTS® is activated to express MMP1 by the oral administration of the small molecule component. The purpose of this open-label single arm Phase 1/2 study is to investigate the safety and effectiveness of FCX-013 plus veledimex.

At Day 0 FCX-013 will be administered intradermally to sclerotic lesions with the possibility of an additional administration pending Week 12 results. Veledimex will be initiated on the day of injection an continue for 2 weeks after the injection of FCX-013.

Genetic: FCX-013

FCX-013 is a genetically modified cell product obtained from the subject's own skin cells (autologous fibroblasts). The cells are expanded and genetically modified to express metalloproteinase-1 (MMP-1) under the control of a RheoSwitch (RTS®) system. FCX-013 cell suspension is injected intradermally.

Other Name: Genetically-Modified Autologous Human Dermal Fibroblasts

Drug: veledimex

Veledimex, is a small molecule which activates the RTS to induce expression of MMP-1 and is and provided as a liquid filled gelatin capsule for oral administration

Other Name: a small-molecule activator ligand for the RheoSwitch (RTS®) system

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information

Ages Eligible for Study:

18 Years and older (Adult, Older Adult)

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

Criteria

Inclusion Criteria:

Adults 18 years or older

Clinical diagnosis of any subtype of localized scleroderma with sclerotic lesions

Subject has not participated in previous clinical research study in the last six months

Subject must be on stable course of systemic immunosuppressive therapy without plan to change or is not on systemic immunosuppressive therapy

Subjects with good control of disease but have sclerotic lesions that have not responded to standard of care

Exclusion Criteria:

Patients with a non-morphea skin disorder in the region of interest

Localized scleroderma/morphea lesions on the face or over a joint, or lesions that can be successfully managed with topical medications or phototherapy